EBMT 2024 | Current SoC for BPDCN and other emerging treatment options
Cristina Papayannidis, MD, PhD, University of Bologna, Bologna, Italy, summarizes the current standard of care (SoC) for patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN). Tagraxofusp, a first-in-class CD123-targeted agent, is particularly efficacious for first-line adult patients with BPDCN. Dr Papayannidis highlights that allogeneic stem cell transplantation (alloSCT) is utilized in virtually all cases of BPDCN following standard chemotherapy regimens. This interview took place at the 50th Annual Meeting of the EBMT in Glasgow, Scotland.
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Transcript (edited for clarity)
So based on the last version of the NCCN guidelines, at diagnosis what we have to do is to identify patients fit for an intensive approach, differently from those who are not candidates to receive an intensive treatment. When we say intensive treatment, we mean a lot of options. But the very first one today is represented by tagraxofusp, which is an anti-CD123 drug which has been approved by FDA, in 2018 and then by EMA in 2020...
So based on the last version of the NCCN guidelines, at diagnosis what we have to do is to identify patients fit for an intensive approach, differently from those who are not candidates to receive an intensive treatment. When we say intensive treatment, we mean a lot of options. But the very first one today is represented by tagraxofusp, which is an anti-CD123 drug which has been approved by FDA, in 2018 and then by EMA in 2020. In particular in Europe, it’s approved for the first line of adult patients with BPDCN.
Other options include the standard chemotherapy based on AML or ALL regimens or non-Hodgkin lymphoma regimens. But what we know is that with standard chemotherapy, unfortunately, the duration of responses are very, very short and only a minority of patients are able to proceed to an allogeneic stem cell transplantation, which is the the final aim of our approach, because, with whatever is the treatment we decide to start, the aim is to achieve a CR and then to proceed to an allogeneic stem cell transplantation, of course for those patients who are fit and eligible for this kind of approach.
Together with these treatments, we have to perform CNS prophylaxis, as I mentioned before, with the lumbar punctures. So at the moment, I must say that the best approach we have in order to achieve the higher rate of CR is tagraxofusp. And based on the data which have been published in the New England, and then in the last follow up in JCO, the overall response rate is 75%.
So the majority of the patients are able to achieve a response.
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Disclosures
Menarini Stemline: Honoraria.
